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UTRs increasing the translation efficiency of RNA molecules

机译:UTR增加了RNA分子的翻译效率

摘要

Described is an RNA molecule comprising (a) a coding region coding for a polypeptide; and (b) upstream of said coding region one or more UTR(s) comprising the sequence as shown in SEQ ID NO:1 or a sequence which shows 1 to 4 substitutions in comparison to SEQ ID NO:1 and which results in an RNA molecule having the same or a higher translation efficiency as an RNA molecule comprising an UTR comprising SEQ ID NO:1; and/or (c) downstream of said coding region one or more UTR(s) comprising the sequence as shown in SEQ ID NO:2 or a sequence which shows 1 to 7 substitutions in comparison to SEQ ID NO:2 and which results in an RNA molecule having the same or a higher translation efficiency as an RNA molecule comprising an UTR comprising SEQ ID NO:2; wherein said polypeptide encoded by said coding region is not a cytochrome b-245 alpha polypeptide (CYBA). Moreover, described is a nucleic acid molecule encoding the RNA molecule according to the present invention. Further, described is a vector comprising the nucleic acid molecule according to the present invention and to a host cell comprising the vector according to the present invention. Further, described is a pharmaceutical composition comprising the RNA molecule according to the present invention and optionally a pharmaceutically acceptable carrier. Moreover, described is a kit comprising the RNA molecule according to the present invention. Finally, described is the use of one or more UTR(s) as defined in (b) and/or one or more UTR(s) as defined in (c) for increasing the efficiency of translating a coding region of an RNA molecule into a polypeptide or a protein encoded by said coding region.
机译:描述是包含(a)编码多肽的编码区的RNA分子; (b)所述编码区域的上游,一种或多种UTR(S),其包含如SEQ ID NO:1的序列或与SEQ ID NO:1相比显示1至4个取代的序列,并导致RNA具有与包含SEQ ID NO:1的UTR的RNA分子相同或更高的翻译效率的分子;和/或(c)所述编码区域的下游,一种或多种UTR(S),其包含如SEQ ID NO:2或与SEQ ID NO:2相比显示1至7个取代的序列的序列,并导致具有与包含SEQ ID NO:2的UTR的RNA分子相同或更高的翻译效率的RNA分子;其中所述编码区编码的所述多肽不是细胞色素B-245α多肽(Cyba)。此外,所述描述根据本发明编码RNA分子的核酸分子。此外,描述的是包含根据本发明的核酸分子和包含根据本发明的载体的宿主细胞的载体。此外,描述的是包含根据本发明的RNA分子和任选的药学上可接受的载体的药物组合物。此外,描述的是包含根据本发明的RNA分子的试剂盒。最后,描述的是使用如(b)中定义的一个或多个UTR(s)和/或如(c)中定义的一个或多个UTR(s)用于增加将RNA分子的编码区域平移到的效率多肽或由所述编码区域编码的蛋白质。

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